A Phase III, Multicenter, Open-Label, Uncontrolled Study To Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, And Pharmacodynamics Of Satralizumab In Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)

Who is this study for? Patients with Neuromyelitis Optica Spectrum Disorder
What treatments are being studied? Satralizumab
Status: Recruiting
Location: See all (9) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 11
Healthy Volunteers: f
View:

• Age at screening 2-11 years, inclusive

• Body weight at screening \>=10 kg

• For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception

• Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria Clinical evidence of at least one documented attack (including first attack) in the last year prior to screening

• Neurological stability for \>=30 days prior to both screening and baseline

• Expanded Disability Status Scale (EDSS) 0 to 6.5

• For patients receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline

Locations
United States
Colorado
Children's Hospital Colorado.
RECRUITING
Denver
Other Locations
Argentina
Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
RECRUITING
Ciudad Autonoma Buenos Aires
Clinica Universitaria Reina Fabiola
RECRUITING
Córdoba
France
Centre Hospitalier Universitaire de Bicêtre
RECRUITING
Le Kremlin-bicêtre
Italy
Fondazione Istituto Neurologico Mondino IRCCS
RECRUITING
Pavia
IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
RECRUITING
Rome
Mexico
Grupo Medico Camino
RECRUITING
Df
Poland
Uniwersyteckie Centrum Kliniczne
ACTIVE_NOT_RECRUITING
Gdansk
United Kingdom
Great Ormond Street Hospital for Children
RECRUITING
London
Contact Information
Primary
Reference Study ID Number: WN41733 https://forpatients.roche.com/
global-roche-genentech-trials@gene.com
888-662-6728 (U.S.)
Backup
Global Medical Information
global.medical_information@roche.com
Time Frame
Start Date: 2025-09-30
Estimated Completion Date: 2029-09-12
Participants
Target number of participants: 8
Treatments
Experimental: Cohort 1: Participants with body weight ≥10kg to <20kg
Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients
Experimental: Cohort 2 Participants with body weight ≥20kg to <40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
Experimental: Cohort 3 Participants with body weight ≥40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter.
Sponsors
Leads: Hoffmann-La Roche

This content was sourced from clinicaltrials.gov